Gene therapy for visual loss: Opportunities and concerns
Jia Hui Lee, Jiang-Hui Wang, Jinying Chen, Fan Li, Thomas L Edwards, Alex W Hewitt, Guei-Sheung Liu
PROGRESS IN RETINAL AND EYE RESEARCH | PERGAMON-ELSEVIER SCIENCE LTD | Published : 2019
Many clinical trials using gene therapy have shown significant therapeutic benefits and exceptional safety records. Increasing evidence is verifying the long sought-after promise that gene therapy will genetically 'cure' some severely disabling diseases. In particular, the first gene therapy bioproduct for RPE65-associated Leber's congenital amaurosis, which was approved by the US Food and Drug Administration in 2017, has provided tremendous encouragement to the field of gene therapy. Recent developments in genome editing technologies have significantly advanced our capability to precisely engineer genomes in eukaryotic cells. Programmable nucleases, particularly the CRISPR/Cas system, have ..View full abstract
Awarded by National Health and Medical Research Council of Australia
Awarded by NHMRC
None of the authors have conflicts of interest to disclose. We thank Professor Dianne Nicol, Ms. Tess Whitton, and Mr. Tucker Lachlan for critically reviewing the manuscript. This work was supported by The National Health and Medical Research Council of Australia (#1061912 and 1123329), The Ophthalmic Research Institute of Australia, The Angior Family Foundation and The Rebecca L. Cooper Medical Research Foundation. J.H.W. received a R.B. McComas Research Scholarship in Ophthalmology, Gordon P. Castles Scholarship and a Melbourne Research Scholarship. A.W.H. received an NHMRC Practitioner Fellowship (#1103329). The Center for Eye Research Australia receives Operational Infrastructure Support from the Victorian Government.